Washington Report: PDUFA Blues

Money makes the world go round—especially in Washington, where the prospect of a few hundred million expendable dollars tends to set off a whirlwind of creativity devoted to spending it. FDA is catching more than its share of the breeze these days. On the one hand, the Prescription Drug User Fee Act has to be reauthorized by October 1, 2007, and all the interested parties are busily fine-tuning their wish lists for "improvements." And, on the other, the Bush administration's plan for pandemic-flu preparedness, which puts billions of dollars on the table to purchase antivirals and vaccines, promises to create an enormous workload for the agency—one that is currently unfunded. The situation poses some threats for FDA. But pharmahas a substantial stake in the PDUFA debate as well—especially if the triple-whammy of new PDUFA-funded mandates, rising workloads, and avian flu initiatives leads to a breakdown in the drug approval process.

Seeking Flexibility

PDUFA season officially opened November 14, at an FDA stakeholder meeting to discuss the reauthorization. Acting commissioner Andrew von Eschenbach and deputy commissioner Janet Woodcock, among others, praised the $250 million program. Although some consumer advocates and their Congressional allies blast user fees, arguing that they boost industry control over the drug approval process, FDA officials, pharma companies, and patient disease groups applaud the program for ending "drug lag" and speeding new therapies to market:

Funding for human drug review rose by more than 225 percent from 1992, when PDUFA first passed, to 2004.

Half of new drugs today are launched in the United States, compared with 8 percent pre-PDUFA.

The program has been expanded twice since 1992 to support post-market surveillance and modernize agency information systems. Now FDA wants more flexibility to use fee revenues to further extend drug-safety oversight and to review DTC advertising before it goes public (an important concern now that companies have pledged under the PhRMA DTC Code to seek such review). The agency also would like to devote some fee revenues to modernizing the ever-longer and more complex drug-development process, as part of its Critical Path initiative (see "What Ever Happened to Critical Path?").

FDA cites a growing workload to justify fee revisions: While NDAs and BLAs are fairly flat, efficacy and manufacturing supplements continue to rise. The agency struggled last year to prepare for more than 2,000 industry-requested meetings and to assess nearly 350 special research protocols for innovations in carcinogenicity testing and clinical trial design, among others.

Similar discussions have begun involving reauthorization of user fees for medical device manufacturers, which also expire in 2007. Officials at FDA's Center for Devices and Radiological Health (CDRH) want to be able to use some of the fees collected under the Medical Device User Fee and Modernization Act (MDUFMA) of 2002 for post-market surveillance, a proposal that has gained momentum following recent safety crises involving pacemakers and defibrillators.

No Christmas Trees

The pharmaceutical industry, for its part, wants to hold the line on fee increases (it currently costs a sponsor nearly $800,000 to file an NDA or BLA) and to restrain the use of fee revenues for activities unrelated to drug development and approval. A broader goal is to prevent a reauthorization bill from becoming a "Christmas tree" loaded with miscellaneous legislation. That's a legitimate concern. Proposals are already circulating on the Hill to establish new drug-safety-oversight arrangements, spur development of follow-on biologics, mandate completion of post-approval studies, and boost oversight of DTC advertising, to name just a few.

There's even an item industry would like to put on the tree: a measure to reauthorize the use of patent extensions as incentive for conducting studies of drugs in pediatric populations, which also is up for renewal in 2007. The six-month extensions have been a boon for pharma companies, while also generating important pediatric labeling information.